4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

Running, not enrolling · Phase 1/Phase 2

Conditions studied: X-Linked Retinitis Pigmentosa

In brief

This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR).

Key facts

Study ID

NCT04517149

Run by

4D Molecular Therapeutics

People needed

21

Starts

2020-06-09

Expected to finish

2029-05-01

Last updated by the study team

2025-03-21

Who can join

Age: 12 and older. Sex: male. Healthy volunteers: not accepted.

Where it is running

• University of Colorado — Aurora, Colorado, United States
• Vitreo Retinal Associates — Gainesville, Florida, United States
• University of Michigan Kellogg Eye Center — Ann Arbor, Michigan, United States
• Columbia University Medical Center/Edward Harkness Eye Institute — New York, New York, United States
• Duke University Eye Center/Dept. of Ophthalmology — Durham, North Carolina, United States
• Casey Eye Institute, Oregon Health and Science University — Portland, Oregon, United States
• Retina Foundation of the Southwest — Dallas, Texas, United States
• University of Utah John A. Moran Eye Center — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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